| Assessment Status | NCPE Assessment Process Complete |
| HTA ID | 21064 |
| Drug | Pegcetacoplan |
| Brand | Aspaveli® |
| Indication | For the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least three months. |
| Assessment Process | |
| Rapid review commissioned | 13/12/2021 |
| Rapid review completed | 26/01/2022 |
| Rapid review outcome | Rapid Review outcome A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of pegcetacoplan compared with the current standard of care, on the basis of the proposed price relative to currently available therapies. |
| Full pharmacoeconomic assessment commissioned by HSE | 23/02/2022 |
| Pre-submission consultation with Applicant | 17/10/2022 |
| Full submission received from Applicant | 26/05/2023 |
| Preliminary review sent to Applicant | 05/12/2023 |
| NCPE assessment re-commenced | 16/01/2024 |
| Follow-up to preliminary review sent to Applicant | 02/02/2024 |
| NCPE assessment re-commenced | 09/02/2024 |
| Factual accuracy sent to Applicant | 08/03/2024 |
| NCPE assessment re-commenced | 19/03/2024 |
| NCPE assessment completed | 24/04/2024 |
| NCPE assessment outcome | The NCPE recommends that pegcetacoplan not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatments*. |
*This recommendation should be considered while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.
